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Clinical Trial News

InspireMD Submits FDA Premarket Approval Application for CGuard Prime Carotid Stent System

  • InspireMD has submitted a Premarket Approval (PMA) application to the FDA for its CGuard Prime carotid stent system, aimed at preventing stroke.
  • The PMA is supported by positive data from the C-GUARDIANS clinical trial, which demonstrated a low major adverse event rate of 1.95% at one year.
  • The C-GUARDIANS trial enrolled 316 patients across U.S. and European sites, evaluating the safety and efficacy of CGuard for carotid artery stenosis.
  • If approved, InspireMD anticipates a U.S. commercial launch of the CGuard Prime system in the first half of 2025.

TransCon CNP Demonstrates Superior Growth in Achondroplasia Trial

  • TransCon CNP met its primary endpoint, showing a statistically significant improvement in annualized growth velocity (AGV) compared to placebo at Week 52 (p<0.0001).
  • Children aged 5-11 years treated with TransCon CNP showed a greater change from baseline AGV compared to placebo, with a statistically significant difference of 1.78 cm/year (p<0.0001).
  • The trial indicated that TransCon CNP was generally well-tolerated, with a low incidence of mild injection site reactions and a safety profile comparable to placebo.
  • Ascendis Pharma plans to submit regulatory applications to the FDA in Q1 2025 and to the EMA in Q3 2025, seeking approval for TransCon CNP as a treatment for achondroplasia.

Imfinzi Demonstrates Significant Survival Benefit in Muscle-Invasive Bladder Cancer

• The NIAGARA trial showed that adding Imfinzi (durvalumab) to neoadjuvant chemotherapy significantly improved event-free survival (EFS) and overall survival (OS) in patients with muscle-invasive bladder cancer (MIBC). • The Imfinzi regimen reduced the risk of death by 25% and improved EFS by 32%, with 82% of patients alive at 24 months compared to 75% in the control group. • The study's results, presented at ESMO, were described as practice-changing, with a manageable toxicity profile, potentially transforming the standard of care for MIBC patients. • AstraZeneca is considering filing the NIAGARA data with the FDA, while the agency previously raised concerns about peri-operative immunotherapy regimens in resectable cancers.

InspireMD Seeks FDA Approval for CGuard Prime Carotid Stent System After Positive Trial Results

• InspireMD has submitted a Premarket Approval application to the FDA for its CGuard Prime carotid stent system, designed to prevent stroke by treating carotid artery stenosis. • The application is based on the C-GUARDIANS clinical trial, which reported a low major adverse event rate of 1.95% at one year post-procedure. • InspireMD is preparing for a potential U.S. commercial launch of CGuard Prime in the first half of 2025, pending FDA approval, and is setting up commercial infrastructure. • The CGuard Prime system utilizes InspireMD's MicroNet technology and aims to become the industry standard for carotid stenting with superior acute and long-term outcomes.

Evaxion's Personalized Cancer Vaccine EVX-01 Shows Promise in Phase 2 Melanoma Trial

  • Evaxion Biotech's EVX-01, combined with pembrolizumab, demonstrates a 69% Overall Response Rate (ORR) in advanced melanoma patients.
  • The personalized vaccine, designed using Evaxion's AI-Immunology platform, induced a targeted immune response in 79% of patients.
  • The AI platform showed a strong predictive capability (p=0.00013) correlating with the observed immune response, suggesting robust technology.
  • Final results from the phase 2 trial are expected in the third quarter of 2025, with potential for significant advancement in melanoma treatment.

FDA Extends Review of Treosulfan NDA for AML and MDS Treatment

  • The FDA has extended the review period for the New Drug Application (NDA) of treosulfan by three months, setting a new PDUFA target action date of January 30, 2025.
  • The extension was prompted by the need for additional time to review supplemental analyses of previously submitted data related to the drug.
  • The NDA seeks approval for treosulfan in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients with acute myeloid leukemia and myelodysplastic syndromes.
  • Medexus remains optimistic about the potential approval of treosulfan and anticipates a commercial launch in the first half of calendar year 2025.

FDA Approves Eli Lilly's Ebglyss (lebrikizumab) for Moderate-to-Severe Atopic Dermatitis

  • The FDA has approved Eli Lilly's Ebglyss (lebrikizumab-lbkz) for treating moderate-to-severe atopic dermatitis in adults and adolescents (12+ years, >=40kg).
  • Ebglyss, a targeted IL-13 inhibitor, is indicated for patients whose atopic dermatitis is not adequately controlled by topical prescription therapies.
  • Clinical trials (ADvocate1, ADvocate2, ADhere) demonstrated that Ebglyss, as monotherapy or with topical corticosteroids, significantly improved skin clearance and itch relief.
  • Ebglyss offers a convenient once-monthly maintenance injection following an initial treatment phase, providing long-lasting symptom relief for eczema patients.

Blenrep Combination Receives Breakthrough Therapy Designation in China for Multiple Myeloma

  • GSK's Blenrep (belantamab mafodotin) combined with bortezomib and dexamethasone (BorDex) has been granted Breakthrough Therapy Designation (BTD) in China for relapsed or refractory multiple myeloma.
  • The designation was based on Phase III DREAMM-7 trial results, which showed statistically significant improvements in progression-free survival (PFS) compared to daratumumab plus BorDex.
  • Multiple myeloma is a growing health concern in China, with approximately 30,000 new cases each year, underscoring the need for novel treatment options.
  • The DREAMM-7 trial demonstrated clinically meaningful improvements across secondary efficacy endpoints, including deeper and more durable responses, with a safety profile consistent with individual agents.

BioNTech to Present Oncology Pipeline Data at ESMO Congress 2024

  • BioNTech will present clinical trial data from its multi-platform oncology pipeline at the ESMO Congress 2024 in Barcelona, Spain, from September 13-17, 2024.
  • The presentations will cover mRNA-based cancer vaccines, next-generation immunomodulators, and targeted therapy approaches, showcasing the breadth of BioNTech's oncology programs.
  • BioNTech believes that combining immunomodulators, targeted therapies, and mRNA-based therapies will drive the future of cancer treatment, according to Prof. Özlem Türeci, M.D.

FDA Lifts Partial Clinical Hold on Zentalis' Azenosertib Studies

• The FDA has lifted the partial clinical hold on Zentalis Pharmaceuticals' azenosertib studies, allowing the company to resume enrollment in ongoing clinical trials. • Zentalis plans to present azenosertib monotherapy data and provide updates on clinical development at a corporate event later this year. • The company remains confident in azenosertib's therapeutic potential for addressing unmet needs in gynecologic malignancies. • Zentalis is on track to meet all previously disclosed data guidance for 2024, including key clinical study results expected in Q4.

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